A Study of NIDO-361 in Patients With SBMA

Analysis of thigh included adductors, hamstrings, and quadriceps and total (whole-body) included muscles in the rotator cuff, arm, torso, and leg muscle regions (38 muscles in total). Muscles were categorized according to their fat content as follows: A-muscles were defined as muscle fat fraction (MFF) < 50% and muscle fat infiltration (MFI) < 10%. A-muscles have a low or slightly elevated fat content to a level that can be present in individuals without a neuromuscular disease. B-muscles were defined as MFF < 50% and MFI ≥ 10%. B-muscles have a muscle fat content at a level where disease involvement is likely. C-muscles were defined as MFF ≥ 50%. C-muscles have a muscle fat content where more than 50% of the muscle tissue has been replaced by fat and most of the functional capacity is likely lost.

Adverse events were coded with MedDRA Dictionary Version 26.1.

Adverse events were coded with MedDRA Dictionary Version 26.1.

The modified Spinal and Bulbar Muscular Atrophy Functional Rating Scale (m-SBMAFRS) is calculated as the sum of the items for the truncal and lower-limb domains from the SBMAFRS, which is a validated functional rating scale developed from the Amyotrophic Lateral Sclerosis Functional Rating Scale. Each item has five responses options scoring 0 (worst) to 4 (normal). A higher score indicates better functioning. Total range is 0 (worst) to 30 (normal).

2-minute walk test (2MWT) measures the distance traveled (meters) in 2 minutes.

6-minute walk test (6MWT) measures the distance traveled (meters) in 6 minutes.

The Timed Up and Go Test (TUG) is used to assess mobility and to provide an estimate on balance and risk of falling. Patients are asked to rise from a chair, walk 3 meters, turn around, return to the chair, and sit. The time that it takes to complete the task is recorded. Assistive devices, orthoses, and ankle braces are allowed.

The Actigraph device is a non-invasive research-grade accelerometer based wearable device that was used to objectively measure free-living physical activity. The data from these devices was used to assess and detect changes in physical activity intensity, upper arm movements, and walking patterns.

The Actigraph device is a non-invasive research-grade accelerometer based wearable device that was used to objectively measure free-living physical activity. The data from these devices was used to assess and detect changes in physical activity intensity, upper arm movements, and walking patterns.

The Actigraph device is a non-invasive research-grade accelerometer based wearable device that was used to objectively measure free-living physical activity. The data from these devices was used to assess and detect changes in physical activity intensity, upper arm movements, and walking patterns.

The Actigraph device is a non-invasive research-grade accelerometer based wearable device that was used to objectively measure free-living physical activity. The data from these devices was used to assess and detect changes in physical activity intensity, upper arm movements, and walking patterns.

The Actigraph device is a non-invasive research-grade accelerometer based wearable device that was used to objectively measure free-living physical activity. The data from these devices was used to assess and detect changes in physical activity intensity, upper arm movements, and walking patterns.

Grip strength (lbs) is averaged across three trials for the right and left hands separately using a handheld dynamometer (HHD).